CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Orkambi (lumacaftor/ivacaftor) for cystic fibrosis — Coverage Criteria

Covers medical necessity and prior authorization requirements for Orkambi for members with cystic fibrosis who meet genotype, age, and other usage criteria. Affects providers prescribing Orkambi and members seeking coverage.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyOrkambi (lumacaftor/ivacaftor) for cystic fibrosis — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization with genetic testing documentation confirming F508del homozygosity and ensure member is ≥1 year and prescriber specialty requirements are met.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

12 monthsauthorization period

>=1 yrmin age

homozygous F508delrequired genotype

Coverage Criteria for Orkambi (lumacaftor/ivacaftor)

Initial Therapy — Authorization of 12 months

Authorization of 12 months may be granted for treatment of cystic fibrosis when all of the following criteria are met:

Cystic Fibrosis Initial Therapy Criteria: A. Genetic testing was conducted to detect a mutation in the CFTR gene.

Genetic testing report required for initial request (see Documentation).

Genotype: B. The member is positive for the F508del mutation on both alleles of the CFTR gene.homozygous F508del

If genotype unknown, use an FDA‑cleared CF mutation test to detect F508del on both alleles.

Age: C. The member is at least 1 year of age.>=1 year

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyOrkambi (lumacaftor/ivacaftor) for cystic fibrosis — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization with genetic testing documentation confirming F508del homozygosity and ensure member is ≥1 year and prescriber specialty requirements are met.

SourceLink

On This Page

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Concomitant Use: D. Orkambi will not be used in combination with other medications containing ivacaftor.

Continuation of Therapy — Authorization of 12 months

Authorization of 12 months may be granted for continued treatment in members requesting reauthorization for an indication listed in Section IV who are experiencing benefit from therapy as evidenced by disease stability or disease improvement (e.g., improvement in FEV1 from baseline).

Continuation Criteria: Member has been previously authorized for an indication listed in the policy and is experiencing benefit from therapy as evidenced by disease stability or disease improvement (e.g., improvement in FEV1 from baseline).

12-month reauthorization period.

The FDA‑approved indication for Orkambi (lumacaftor/ivacaftor) is for treatment of cystic fibrosis in patients who are homozygous for the F508del CFTR mutation. Efficacy and safety have been established only in this population; if genotype is unknown, use an FDA‑cleared CF mutation test to confirm the presence of F508del on both alleles.

Use of Orkambi for any indication other than cystic fibrosis in patients who are homozygous for the F508del mutation is considered not medically necessary because the efficacy and safety have not been established; such uses are treated as experimental/investigational.

What Providers Must Do

Prior Authorization

Prior Authorization Required

Prior authorization is required. Initial authorization may be granted for up to 12 months when all clinical criteria are met, including documented genetic testing confirming homozygosity for the F508del CFTR mutation and age requirements.

Initial authorization up to 12 months

Denial Risk

Non‑Covered Indications

Requests for use of Orkambi (lumacaftor/ivacaftor) for indications other than cystic fibrosis in patients homozygous for the F508del CFTR mutation are considered experimental/investigational and not medically necessary.

Documentation Required

Required Documentation

For initial prior authorization requests, submit a genetic testing report documenting the presence of the F508del mutation on both alleles of the CFTR gene. Also include documentation of patient age to confirm they are ≥ 1 year.

Genetic testing report confirming F508del homozygosity (both alleles)
Documentation of patient age (≥ 1 year)
If genotype unknown, an FDA‑cleared CF mutation test should be used

Note

Concomitant Therapy Restriction

Orkambi must not be used in combination with other medications that contain ivacaftor.

Background

Orkambi is a combination therapy of lumacaftor and ivacaftor indicated for the treatment of cystic fibrosis in patients aged 1 year and older who are homozygous for the F508del CFTR mutation. The policy follows the FDA‑approved age limit and requires genotype confirmation for appropriate use.

Definitions

F508del homozygous

DefinitionPresence of the F508del mutation on both alleles of the CFTR gene as detected by an FDA-cleared CF mutation test.

Clinical contextRequired genotype for Orkambi: homozygous F508del CFTR mutation (F508del on both alleles).

Testing noteIf genotype is unknown, use an FDA‑cleared CF mutation test to detect F508del on both alleles.

Implication for coverageEfficacy and safety of Orkambi have not been established for patients with CF who do not have homozygous F508del; other indications are experimental/investigational and not medically necessary.